Sabharwal Priyanka, Wisniewski Thomas
Department of Neurology, New York University School of Medicine, New York, USA.
Department of Neurology, New York University School of Medicine, New York, USA ; Department of Pathology, New York University School of Medicine, New York, USA ; Department of Psychiatry, New York University School of Medicine, New York, USA.
Zhongguo Xian Dai Shen Jing Ji Bing Za Zhi. 2014;14(3):139-151. doi: 10.3969/j.issn.1672-6731.2014.03.001.
Alzheimer's disease (AD), the most prevalent form of dementia worldwide, can be deemed as the next global health epidemic. The biochemistry underlying deposition of amyloid beta (A β) and hyperphosphorylated tau aggregates in AD has been extensively studied. The oligomeric forms of A β that are derived from the normal soluble A β peptides are believed to be the most toxic. However, it is the fibrillar Aβ form that aggregates as amyloid plaques and cerebral amyloid angiopathy, which serve as pathological hallmarks of AD. Moreover, deposits of abnormally phosphorylated tau that form soluble toxic oligomers and then accumulate as neurofibrillary tangles are an essential part of AD pathology. Currently, many strategies are being tested that either inhibit, eradicate or prevent the development of plaques in AD. An exciting new approach on the horizon is the immunization approach. Dramatic results from AD animal models have shown promise for active and passive immune therapies targeting A β. However, there is very limited data in humans that suggests a clear benefit. Some hurdles faced with these studies arise from complications noted with therapy. Encephalitis has been reported in trials of active immunization and vasogenic edema or amyloid - related imaging abnormalities (ARIA) has been reported with passive immunization in a minority of patients. As yet, therapies targeting only tau are still limited to mouse models with few studies targeting both pathologies. As the majority of approaches tried so far are based on targeting a self - protein, though in an abnormal conformation, benefits of therapy need to be balanced against the possible risks of stimulating excessive toxic inflammation. For better efficacy, future strategies will need to focus on the toxic oligomers and targeting all aspects of AD pathology.
阿尔茨海默病(AD)是全球最常见的痴呆形式,可被视为下一场全球健康危机。AD中淀粉样β蛋白(Aβ)沉积和过度磷酸化tau蛋白聚集体形成的生物化学机制已得到广泛研究。源自正常可溶性Aβ肽的Aβ寡聚体形式被认为毒性最强。然而,正是纤维状Aβ形式聚集成淀粉样斑块和脑淀粉样血管病变,这些是AD的病理特征。此外,异常磷酸化的tau蛋白沉积物形成可溶性毒性寡聚体,然后积聚为神经原纤维缠结,是AD病理的重要组成部分。目前,许多策略正在进行测试,这些策略要么抑制、根除AD中的斑块,要么预防其发展。即将出现的一种令人兴奋的新方法是免疫疗法。AD动物模型的显著结果显示了针对Aβ的主动和被动免疫疗法的前景。然而,在人类中仅有非常有限的数据表明有明显益处。这些研究面临的一些障碍源于治疗中出现的并发症。在主动免疫试验中报告了脑炎,在少数接受被动免疫的患者中报告了血管源性水肿或淀粉样蛋白相关成像异常(ARIA)。到目前为止,仅针对tau蛋白的疗法仍仅限于小鼠模型,针对两种病理的研究很少。由于迄今为止尝试的大多数方法都是基于靶向一种自身蛋白,尽管其构象异常,但治疗的益处需要与刺激过度毒性炎症的潜在风险相权衡。为了提高疗效,未来的策略需要关注毒性寡聚体并针对AD病理的各个方面。